Rare Disease Case Studies

Author(s)

Mariya Kamusheva, M.Ph, PhD, Medical University of Sofia, Sofia, 22, Bulgaria

These case studies include improving patient care for Multiple Sclerosis through proximity-oriented approaches in Italy, utilizing real-world evidence for health technology assessment in sickle cell disease in the UK, using patient registries to inform HTA in rare diseases in Brazil, and addressing challenges in using registries for research and patient outcomes in European Haemophilia Consortium.

Conference/Value in Health Info

2023-11, ISPOR Europe 2023, Copenhagen, Denmark

Value in Health, Volume 26, Issue 11, S2 (December 2023)

Code

318

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